The U.S. Food and Drug Administration (FDA) has approved Samsung Bioepis’ Biologics License Application (BLA) for EPYSQLI (eculizumab-aagh) as a biosimilar to Soliris (eculizumab).
EPYSQLI has been approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. EPYSQLI is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
The FDA’s approval of EPYSQLI is based on a totality of evidence including analytical, non-clinical and clinical data demonstrating it is highly similar to Soliris, with no clinically meaningful differences between EPYSQLI and Soliris in terms of safety, purity and potency: The randomized Phase I, double-blind, three-arm, parallel group, single-dose study in healthy volunteers demonstrated pharmacokinetics (PK) equivalence and comparable pharmacodynamic (PD), safety, tolerability, and immunogenicity profiles between EPYSQLI and Soliris. The randomized Phase 3, double-blind, multicenter, cross-over study in PNH patients demonstrated clinical equivalence in efficacy, safety, PK, and immunogenicity between EPYSQLI and Soliris.
“The FDA approval of EPYSQLI as a biosimilar to Soliris marks an important milestone for PNH and aHUS communities since biosimilars have a potential to positively impact patients and healthcare systems by reducing healthcare costs and improving access to treatments,” said Christopher Hansung Ko, President and Chief Executive Officer at Samsung Bioepis.
“Our mission has been, and always will be improving the lives of patients by providing quality-assured, safe and effective biologic medicines, and our work to fulfill this mission is expanding into rare disease areas where patients continue to suffer from limited access to life-enhancing medicines,” he added.
The monoclonal antibody and anti-C5 complement inhibitor eculizumab is a well-established standard treatment for PNH and aHUS, rare diseases with an estimated US prevalence of approximately 50,000 and 5,000 respectively. Approximately 70% of eculizumab-treated PNH patients are not dosed according to the label, and two-thirds of patients discontinue eculizumab within an average of 1.5 years, which may be attributed to several factors including the high treatment cost.
Biosimilars, biologic medicines that are highly similar to and have no clinically meaningful differences from an existing FDA-approved biologic, have the potential to relieve the financial burden of the healthcare system and improve access to biologic therapies. Biosimilar drugs must meet the same standards for pharmaceutical quality, safety and efficacy as reference products.
Outside of the US, EPYSQLI has also been approved by the European Commission (EC) and Korea’s Ministry of Food and Drug Safety (MFDS) as a biosimilar to Soliris for the treatment of patients with PNH and aHUS.
